Cited by 214RNA Interference and Gene DeliveryVirus-based gene therapy researchAnimal Genetics and Reproduction

The DOI matches its Crossref record; 34 of the 34 references it cites were found in a registry or an index.

Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor1α promoter

Gill, DR, Smyth, SE, Goddard, CA, Pringle, IA, Higgins, CF, Colledge, WH, Hyde, SC·2001·Gene Therapy

Open this paper in CoChat — free account

Abstract

For effective gene therapy of chronic disease, persistent transgene expression at therapeutic levels is required. Clinical studies of airway gene transfer in patients with cystic fibrosis (CF) have resulted in short-lived transgene expression. We used intra-nasal dosing of naked plasmid DNA to the murine lung as a model for investigating the duration of airway gene transfer from a series of reporter expression plasmids. Transgene expression was transient when mediated by the viral promoters CMV, RSV and SV40, falling to less than 10% of peak expression after 2 weeks, although the presence of the adenoviral E4ORF3 gene in cis, resulted in extended duration of reporter activity from the CMV promoter. Transient expression from these promoters was not due to loss of the vector as determined by quantitative TaqMan PCR analysis. However, use of the promoters from the human polybiquitin C (UbC) and the elongation factor 1alpha (EF1alpha) genes resulted in persistent gene expression in the mouse lung. The UbC promoter directed high-level reporter activity which was maintained for up to 8 weeks and was still detectable 6 months after a single administration. Such persistent airway transgene expression from a nonviral vector without the concomitant expression of a potential antigen has not been reported previously. Thus, despite the persistence of vector DNA in vivo, attenuation of promoter function may lead to silencing of transgene expression and careful selection of promoter sequenc…

Integrity signals

Registry

Crossref · 10.1038/sj.gt.3301561

Cites

34 of 34 references verified0 citation errors · 0 not checkable

Statistics

Recomputing p-values needs the paper’s full text.

Claims the paper makes

This paper’s full text isn’t openly available, so its claims and self-check weren’t run. The registry and citation checks still apply.

Claims other papers make about this one

Each is checked against this paper’s own text: does it say what the citing paper says it does?

Characteristics of a human cell line transformed by DNA from human adenovirus type 5
Graham, F. L., Russell, W. C., Smiley, J., & Nairn, R. (1977) · Journal of General Virology · doi:10.1099/0022-1317-36-1-59 · Cited by 4,793
Point mutations define a sequence flanking the AUG initiator codon that modulates translation by eukaryotic ribosomes
Kozak, M. (1986) · Cell · doi:10.1016/0092-8674(86)90762-2 · Cited by 4,741
A novel cationic liposome reagent for efficient transfection of mammalian cells
Gao, X., & Huang, L. (1991) · Biochemical and Biophysical Research Communications · doi:10.1016/0006-291x(91)91366-k · Cited by 847
Detailed Analysis of Structures and Formulations of Cationic Lipids for Efficient Gene Transfer to the Lung
Lee, E. R., Marshall, J., Siegel, C., Jiang, C., Yew, N. S., Nichols, M., Nietupski, J. B., Ziegler, R. J., Lane, M. B., Wang, K. X., et al. (1996) · Human Gene Therapy · doi:10.1089/hum.1996.7.14-1701 · Cited by 443
DNA Methylation and Silencing of Gene Expression
Newell-Price, J. D. C., Clark, A. J. L., & King, P. (2000) · Trends in Endocrinology and Metabolism · doi:10.1016/s1043-2760(00)00248-4 · Cited by 330
Promoter Attenuation in Gene Therapy: Interferon- γ and Tumor Necrosis Factor- α Inhibit Transgene Expression
Qin, L., Ding, Y., Pahud, D. R., Chang, E., Imperiale, M. J., & Bromberg, J. S. (1997) · Human Gene Therapy · doi:10.1089/hum.1997.8.17-2019 · Cited by 315
Role of covalent modifications of histones in regulating gene expression
Spencer, V. A., & Davie, J. (1999) · Gene · doi:10.1016/s0378-1119(99)00405-9 · Cited by 306
Reactivation of the Previously Silenced Cytomegalovirus Major Immediate-Early Promoter in the Mouse Liver: Involvement of NFκB
Löser, P., Jennings, G. S., Strauss, M., & Sandig, V. (1998) · Journal of Virology · doi:10.1128/jvi.72.1.180-190.1998 · Cited by 277
Isolation and Characterization of the Human Chromosomal Gene for Polypeptide Chain Elongation Factor-1α
Uetsuki, T., Naito, A., Nagata, S., & Kaziro, Y. (1989) · Journal of Biological Chemistry · doi:10.1016/s0021-9258(18)83619-5 · Cited by 272
Targeting of adenovirus E1A and E4-ORF3 proteins to nuclear matrix-associated PML bodies.
Carvalho, T., Seeler, J.-S., Öhman, K., Jordan, P., Pettersson, U., Akusjärvi, G., Carmo‐Fonseca, M., & Dejean, A. (1995) · The Journal of Cell Biology · doi:10.1083/jcb.131.1.45 · Cited by 267

Show all 34 references

Sorted by citations

Non-viral vectors for gene-based therapy
Yin, H., Kanasty, R. L., Eltoukhy, A. A., Vegas, A. J., Dorkin, J. R., & Anderson, D. G. (2014) · Nature Reviews Genetics · doi:10.1038/nrg3763 · Cited by 3,123
Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo
Chen, Z.-Y., He, C.-Y., Ehrhardt, A., & Kay, M. A. (2003) · Molecular Therapy · doi:10.1016/s1525-0016(03)00168-0 · Cited by 463
Recombinant protein production by large-scale transient gene expression in mammalian cells: state of the art and future perspectives
Baldi, L., Hacker, D. L., Adam, M., & Wurm, F. M. (2007) · Biotechnology Letters · doi:10.1007/s10529-006-9297-y · Cited by 328
CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression
Hyde, S. C., Pringle, I. A., Abdullah, S., Lawton, A. E., Davies, L. A., Varathalingam, A., Nunez-Alonso, G. A., Green, A.-M., Bazzani, R. P., Jones, S., et al. (2008) · Nature Biotechnology · doi:10.1038/nbt1399 · Cited by 308
Self-citation
Compacted DNA Nanoparticles Administered to the Nasal Mucosa of Cystic Fibrosis Subjects Are Safe and Demonstrate Partial to Complete Cystic Fibrosis Transmembrane Regulator Reconstitution
Konstan, M. W., Davis, P. B., Wagener, J. S., Hilliard, K. A., Stern, R. C., Milgram, L. J. H., Kowalczyk, T. H., Hyatt, S. L., Fink, T. L., Gedeon, C. R., et al. (2004) · Human Gene Therapy · doi:10.1089/hum.2004.15.1255 · Cited by 298
Functional, Persistent, and Extended Liver to Pancreas Transdifferentiation
Ber, I., Keren Shternhall-ron, A., Perl, S., Ohanuna, Z., Goldberg, I., Barshack, I., Benvenisti-Zarum, L., Meivar‐Levy, I., & Ferber, S. (2003) · Journal of Biological Chemistry · doi:10.1074/jbc.m303127200 · Cited by 264
Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo
Chen, Z. Y., He, C. Y., Meuse, L. A., & Kay, M. A. (2004) · Gene Therapy · doi:10.1038/sj.gt.3302231 · Cited by 232
Adeno-Associated Virus Technologies and Methods for Targeted Neuronal Manipulation
Haery, L., Deverman, B. E., Matho, K. S., Cetin, A. H., Woodard, K., Cepko, C. L., Guerin, K. I., Rego, M. A., Ersing, I., Bächle, S. M., et al. (2019) · Frontiers in Neuroanatomy · doi:10.3389/fnana.2019.00093 · Cited by 220
Developing MR reporter genes: promises and pitfalls
Gilad, A. A., Winnard, P. T., van Zijl, P. C. M., & Bulte, J. W. M. (2007) · NMR in Biomedicine · doi:10.1002/nbm.1134 · Cited by 189
Gene therapy progress and prospects: cystic fibrosis
Uta Griesenbach, D., Geddes, D. M., & Alton, E. W. F. W. (2006) · Gene Therapy · doi:10.1038/sj.gt.3302809 · Cited by 174
Promoters and Control Elements: Designing Expression Cassettes for Gene Therapy
Papadakis, E. D., Nicklin, S. A., Baker, A. H., & White, S. J. (2004) · Current Gene Therapy · doi:10.2174/1566523044578077 · Cited by 175
A toolkit for Nannochloropsis oceanica CCMP 1779 enables gene stacking and genetic engineering of the eicosapentaenoic acid pathway for enhanced long‐chain polyunsaturated fatty acid production
Poliner, E., Pulman, J. A., Zienkiewicz, K., Childs, K. L., Benning, C., & Farré, E. M. (2017) · Plant Biotechnology Journal · doi:10.1111/pbi.12772 · Cited by 169
Barriers to and new approaches for gene therapy and gene delivery in cystic fibrosis
Ferrari, S., Geddes, D. M., & Alton, E. W. F. W. (2002) · Advanced Drug Delivery Reviews · doi:10.1016/s0169-409x(02)00145-x · Cited by 166
Silencing of Episomal Transgene Expression in Liver by Plasmid Bacterial Backbone DNA Is Independent of CpG Methylation
Chen, Z.-Y., Riu, E., He, C.-Y., Xu, H., & Kay, M. A. (2008) · Molecular Therapy · doi:10.1038/sj.mt.6300399 · Cited by 160
Non-Invasive Detection of a Small Number of Bioluminescent Cancer Cells In Vivo
Kim, J., Urban, K., Cochran, E. D., Lee, S., Ang, A., Rice, B., Bata, A., Campbell, K. D., Coffee, R. J., Gorodinsky, A., et al. (2010) · PLoS ONE · doi:10.1371/journal.pone.0009364 · Cited by 159
Gene transfer to the lung: Lessons learned from more than 2 decades of CF gene therapy
Uta Griesenbach, D., & Alton, E. W. F. W. (2008) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2008.09.010 · Cited by 164
Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis
Alton, E. W. F. W., Beekman, J. M., Boyd, A. C., Brand, J., Carlon, M. S., Connolly, M. M., Chan, M., Conlon, S., Davidson, H. E., Davies, J. C., et al. (2017) · Thorax · doi:10.1136/thoraxjnl-2016-208406 · Cited by 150
Self-citation
Multilineage Cells from Adipose Tissue as Gene Delivery Vehicles
Morizono, K., Ugarte, D. A. D., Zhu, M. P., Zuk, P., Elbarbary, A. S., Ashjian, P. H., Benhaim, P., Chen, I. S. Y., & Hedrick, M. H. (2003) · Human Gene Therapy · doi:10.1089/10430340360464714 · Cited by 146
Viral Expression Cassette Elements to Enhance Transgene Target Specificity and Expression in Gene Therapy
The University of North Carolina at Chapel Hill University Libraries (2015) · PubMed · doi:10.17615/2x9m-k248 · Cited by 159
DNA vaccines: improving expression of antigens
Garmory, H. S., Brown, K. A., & Titball, R. W. (2003) · Genetic Vaccines and Therapy · doi:10.1186/1479-0556-1-2 · Cited by 143
Intranasal gene delivery with a polyethylenimine–PEG conjugate
Kichler, A., Chillón, M., Leborgne, C., Danos, O., & Frisch, B. (2002) · Journal of Controlled Release · doi:10.1016/s0168-3659(02)00080-9 · Cited by 131
Systemic delivery of mRNA and DNA to the lung using polymer-lipid nanoparticles
Kaczmarek, J. C., Patel, A. K., Rhym, L. H., Palmiero, U. C., Bhat, B., Heartlein, M. W., DeRosa, F., & Anderson, D. G. (2021) · Biomaterials · doi:10.1016/j.biomaterials.2021.120966 · Cited by 110
Enhancement of plasmid-mediated gene therapy for muscular dystrophy by directed plasmid integration
Bertoni, C., Jarrahian, S., Wheeler, T. M., Li, Y., Olivares, E. C., Calos, M. P., & Rando, T. A. (2005) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0504505102 · Cited by 107
Gene insertion and long-term expression in lung mediated by the sleeping beauty transposon system
Belur, L. R., Frandsen, J. L., Dupuy, A. J., Ingbar, D. H., Largaespada, D. A., Hackett, P. B., & McIvor, R. S. (2003) · Molecular Therapy · doi:10.1016/s1525-0016(03)00211-9 · Cited by 105
Pre-clinical evaluation of three non-viral gene transfer agents for cystic fibrosis after aerosol delivery to the ovine lung
McLachlan, G., Davidson, H. M., Holder, E. L., Davies, L. A., Pringle, I. A., Jones, S., Baker, A., Tennant, P. A., Gordon, C., Vrettou, C., et al. (2011) · Gene Therapy · doi:10.1038/gt.2011.55 · Cited by 104
Self-citation
Scanning surface confocal microscopy for simultaneous topographical and fluorescence imaging: Application to single virus-like particle entry into a cell
Gorelik, J., Shevchuk, A., Ramalho, M., Elliott, M., Lei, C., Higgins, C. F., Lab, M. J., Klenerman, D., Krauzewicz, N., & Korchev, Y. E. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.252458399 · Cited by 103
Self-citation
Novel Minicircle Vector for Gene Therapy in Murine Myocardial Infarction
Huang, M., Chen, Z., Hu, S., Jia, F., Li, Z., Hoyt, G. E., Robbins, R. C., Kay, M. A., & Wu, J. C. (2009) · Circulation · doi:10.1161/circulationaha.108.841155 · Cited by 99
Induction of effective therapeutic antitumor immunity by direct in vivo administration of lentiviral vectors
Dullaers, M., Meirvenne, S. V., Heirman, C., Straetman, L., Bonehill, A., Aerts, J. L., Thielemans, K., & Breckpot, K. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302697 · Cited by 102
piggyBac Transposon-mediated Long-term Gene Expression in Mice
Nakanishi, H., Higuchi, Y., Kawakami, S., Yamashita, F., & Hashida, M. (2010) · Molecular Therapy · doi:10.1038/mt.2009.302 · Cited by 97
Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays
Buck, T., & Wijnholds, J. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms21124197 · Cited by 92
“(TATA box, Inr, MTE and DPE) [48] EF1-α 2500 Ubiquitous, ++ [51,70] PGK 426 Ubiquitous, ++ [53] UbC 403 Ubiquitous, ++ [70]”
Gene transfer of hepatocyte growth factor by electroporation reduces bleomycin-induced lung fibrosis
Gazdhar, A., Fachinger, P., van Leer‐Buter, C., Pieróg, J., Gugger, M., Friis, R. R., Schmid, R. A., & Geiser, T. (2006) · American Journal of Physiology-Lung Cellular and Molecular Physiology · doi:10.1152/ajplung.00082.2006 · Cited by 92
Novel molecular approaches to cystic fibrosis gene therapy
LEE, T. W. R., Matthews, D. A., & Blair, G. E. (2005) · Biochemical Journal · doi:10.1042/bj20041923 · Cited by 90
CMV enhancer/human PDGF-β promoter for neuron-specific transgene expression
Liu, B., Wang, X., Ma, Y. X., & Wang, S. (2003) · Gene Therapy · doi:10.1038/sj.gt.3302126 · Cited by 89
Homogeneity and persistence of transgene expression by omitting antibiotic selection in cell line isolation
Kaufman, W. L., Kocman, I., Agrawal, V., Rahn, H.-P., Besser, D., & Gossen, M. (2008) · Nucleic Acids Research · doi:10.1093/nar/gkn508 · Cited by 93
Using magnetic forces to enhance non-viral gene transfer to airway epithelium in vivo
Xenariou, S., Uta Griesenbach, D., Ferrari, S., Dean, P., Scheule, R. K., Cheng, S. H., Geddes, D. M., Plank, C., & Alton, E. W. F. W. (2006) · Gene Therapy · doi:10.1038/sj.gt.3302803 · Cited by 87
Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing
Maule, G., Casini, A., Montagna, C., Ramalho, A. S., De Boeck, K., Debyser, Z., Carlon, M. S., Petris, G., & Cereseto, A. (2019) · Nature Communications · doi:10.1038/s41467-019-11454-9 · Cited by 86
The EF‐1α promoter maintains high‐level transgene expression from episomal vectors in transfected CHO‐K1 cells
Wang, X.-Y., Xu, Z.-J., Tian, Z., Zhang, X., Xu, D., Li, Q., Zhang, J., & Wang, T. (2017) · Journal of Cellular and Molecular Medicine · doi:10.1111/jcmm.13216 · Cited by 84
Long-term Transgene Expression in the Central Nervous System Using DNA Nanoparticles
Yurek, D. M., Fletcher, A. M., Smith, G. M., Seroogy, K. B., Ziady, A. G., Molter, J. P., Kowalczyk, T. H., Padegimas, L., & Cooper, M. J. (2009) · Molecular Therapy · doi:10.1038/mt.2009.2 · Cited by 79
Enhanced Lung Gene Expression After Aerosol Delivery of Concentrated pDNA/PEI Complexes
Davies, L. A., McLachlan, G., Jones, S., Ferguson, D. J. P., Baker, A., Tennant, P. A., Gordon, C., Vrettou, C., Baker, E., Zhu, J., et al. (2008) · Molecular Therapy · doi:10.1038/mt.2008.96 · Cited by 79
Self-citation
Postintegrative Gene Silencing within the Sleeping Beauty Transposition System
Garrison, B. S., Yant, S. R., Mikkelsen, J. G., & Kay, M. A. (2007) · Molecular and Cellular Biology · doi:10.1128/mcb.00498-07 · Cited by 78
Moving forward: cystic fibrosis gene therapy
Uta Griesenbach, D., & Alton, E. W. F. W. (2013) · Human Molecular Genetics · doi:10.1093/hmg/ddt372 · Cited by 76
Plasmid DNA Sequences Present in Conventional Herpes Simplex Virus Amplicon Vectors Cause Rapid Transgene Silencing by Forming Inactive Chromatin
Suzuki, M., Kasai, K., & Saeki, Y. (2006) · Journal of Virology · doi:10.1128/jvi.80.7.3293-3300.2006 · Cited by 75
Compacted DNA Nanoparticle Gene Transfer of GDNF to the Rat Striatum Enhances the Survival of Grafted Fetal Dopamine Neurons
Yurek, D. M., Fletcher‐Turner, A., Kowalczyk, T. H., Padegimas, L., & Cooper, M. J. (2009) · Cell Transplantation · doi:10.3727/096368909x12483162196881 · Cited by 74
Efficient suppression of endogenous CFTR nonsense mutations using anticodon-engineered transfer RNAs
Ko, W., Porter, J. J., Sipple, M. T., Edwards, K. M., & Lueck, J. D. (2022) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2022.04.033 · Cited by 72
Minicircle DNA Provides Enhanced and Prolonged Transgene Expression Following Airway Gene Transfer
Munye, M. M., Tagalakis, A. D., Barnes, J. L., Brown, R. E., McAnulty, R. J., Howe, S. J., & Hart, S. L. (2016) · Scientific Reports · doi:10.1038/srep23125 · Cited by 72
Gene therapy for cystic fibrosis lung disease: current status and future perspectives.
Rosenecker, J., Huth, S., & Rudolph, C. (2006) · PubMed · Cited by 72
Therapeutic promise of engineered nonsense suppressor tRNAs
Porter, J. J., Heil, C. S., & Lueck, J. D. (2021) · Wiley Interdisciplinary Reviews - RNA · doi:10.1002/wrna.1641 · Cited by 73
Polyethylenimine-mediated gene delivery to the lung and therapeutic applications
Conese, M. (2008) · Drug Design Development and Therapy · doi:10.2147/dddt.s2708 · Cited by 71
Dlk1/FA1 Is a Novel Endocrine Regulator of Bone and Fat Mass and Its Serum Level Is Modulated by Growth Hormone
Abdallah, B. M., Ding, M. L., Jensen, C. H., Ditzel, N., Flyvbjerg, A., Jensen, T. G., Dagnæs‐Hansen, F., Gasser, J. A., & Kassem, M. S. (2007) · Endocrinology · doi:10.1210/en.2007-0171 · Cited by 69
Gene and cell therapy for cystic fibrosis: From bench to bedside
Conese, M., Ascenzioni, F., Boyd, A., Coutelle, C., Fino, I. D., Smedt, S. C. D., Rejman, J., Rosenecker, J., Schindelhauer, D., & Scholte, B. J. (2011) · Journal of Cystic Fibrosis · doi:10.1016/s1569-1993(11)60017-9 · Cited by 71
Development of S/MAR minicircles for enhanced and persistent transgene expression in the mouse liver
Argyros, O., Wong, S. P., Fedonidis, C., Tolmachov, O. E., Waddington, S. N., Howe, S. J., Niceta, M., Coutelle, C. C., & Harbottle, R. P. (2011) · Journal of Molecular Medicine · doi:10.1007/s00109-010-0713-3 · Cited by 68
Nonviral gene transfer to skeletal, smooth, and cardiac muscle in living animals
Dean, D. A. (2005) · American Journal of Physiology-Cell Physiology · doi:10.1152/ajpcell.00613.2004 · Cited by 67
A potent enhancer element in the 5′-UTR intron is crucial for transcriptional regulation of the human ubiquitin C gene
Bianchi, M., Crinelli, R., Giacomini, E., Carloni, E., & Magnani, M. (2009) · Gene · doi:10.1016/j.gene.2009.08.013 · Cited by 70
Gene Therapy Progress and Prospects: Cystic fibrosis
Uta Griesenbach, D., Ferrari, S., Geddes, D. M., & Alton, E. W. F. W. (2002) · Gene Therapy · doi:10.1038/sj.gt.3301791 · Cited by 67
Effect of the content of unmethylated CpG dinucleotides in plasmid DNA on the sustainability of transgene expression
Mitsui, M., Nishikawa, M., Zang, L., Ando, M., Hattori, K., Takahashi, Y., Watanabe, Y., & Takakura, Y. (2009) · The Journal of Gene Medicine · doi:10.1002/jgm.1317 · Cited by 63
Genetic modification of hematopoietic stem cells with nonviral systems: past progress and future prospects
Papapetrou, E. P., Zoumbos, N. C., & Athanassiadou, A. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302626 · Cited by 63
Impact of Using Different Promoters and Matrix Attachment Regions on Recombinant Protein Expression Level and Stability in Stably Transfected CHO Cells
Ho, S. C. L., Mariati, Yeo, J. H. M., Fang, S. G., & Yang, Y. (2014) · Molecular Biotechnology · doi:10.1007/s12033-014-9809-2 · Cited by 62
Use of ultrasound to enhance nonviral lung gene transfer in vivo
Xenariou, S., Uta Griesenbach, D., Liang, H.-D., Zhu, J., Farley, R., Somerton, L., Singh, C., Jeffery, P. K., Ferrari, S., Scheule, R. K., et al. (2007) · Gene Therapy · doi:10.1038/sj.gt.3302922 · Cited by 58
Gene therapy for cystic fibrosis: an example for lung gene therapy
Uta Griesenbach, D., Geddes, D. M., & Alton, E. W. F. W. (2004) · Gene Therapy · doi:10.1038/sj.gt.3302368 · Cited by 57
Aerosol Delivery of DNA/Liposomes to the Lung for Cystic Fibrosis Gene Therapy
Davies, L. A., Nunez-Alonso, G. A., McLachlan, G., Hyde, S. C., & Gill, D. R. (2014) · Human Gene Therapy Clinical Development · doi:10.1089/hum.2014.019 · Cited by 56
Self-citation
Passport , a native Tc1 transposon from flatfish, is functionally active in vertebrate cells
Clark, K. J., Carlson, D. F., Leaver, M. J., Foster, L. K., & Fahrenkrug, S. C. (2009) · Nucleic Acids Research · doi:10.1093/nar/gkn1025 · Cited by 56
A Phase I/IIa Safety and Efficacy Study of Nebulized Liposome-mediated Gene Therapy for Cystic Fibrosis Supports a Multidose Trial
Alton, E. W. F. W., Boyd, A., Porteous, D. J., Davies, G., Davies, J. C., Uta Griesenbach, D., Higgins, T., Gill, D. R., Hyde, S. C., & Innes, J. A. (2015) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201506-1193le · Cited by 61
Self-citation
In Vivo Electroporation Mediated Gene Delivery to the Beating Heart
Ayuni, E., Gazdhar, A., Giraud, M., Kadner, A., Gugger, M., Cecchini, M. G., Caus, T., Carrel, T. P., Schmid, R. A., & Tevaearai, H. T. (2010) · PLoS ONE · doi:10.1371/journal.pone.0014467 · Cited by 55
Enhancement of airway gene transfer by DNA nanoparticles using a pH-responsive block copolymer of polyethylene glycol and poly-l-lysine
Boylan, N. J., Kim, A. J., Suk, J. S., Adstamongkonkul, P., Simons, B. W., Lai, S. K., Cooper, M. J., & Hanes, J. S. (2011) · Biomaterials · doi:10.1016/j.biomaterials.2011.11.080 · Cited by 54
The SP-C promoter facilitates alveolar type II epithelial cell-specific plasmid nuclear import and gene expression
DeGiulio, J. V., Kaufman, C. D., & Dean, D. A. (2010) · Gene Therapy · doi:10.1038/gt.2009.166 · Cited by 52
The contribution of plasmid design and release to in vivo gene expression following delivery from cationic polymer modified scaffolds
Avilés, M. O., Lin, C.-H., Zelivyanskaya, M. L., Graham, J. G., Boehler, R. M., Messersmith, P. B., & Shea, L. D. (2009) · Biomaterials · doi:10.1016/j.biomaterials.2009.10.035 · Cited by 52
Evaluation of viral and mammalian promoters for driving transgene expression in mouse liver
Al-Dosari, M. S., Zhang, G., Knapp, J. E., & Liu, D. (2005) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2005.11.063 · Cited by 52
New organs from our own tissues: liver-to-pancreas transdifferentiation
Meivar‐Levy, I., & Ferber, S. (2003) · Trends in Endocrinology and Metabolism · doi:10.1016/j.tem.2003.10.006 · Cited by 52
From pathogen to medicine: HIV-1-derived lentiviral vectors as vehicles for dendritic cell based cancer immunotherapy
Dullaers, M., & Thielemans, K. (2005) · The Journal of Gene Medicine · doi:10.1002/jgm.846 · Cited by 52
Morphological Analysis and Lentiviral Transduction of Fetal Monkey Bone Marrow-Derived Mesenchymal Stem Cells
Lee, C. I., Kohn, D. B., Ekert, J. E., & Tarantal, A. F. (2003) · Molecular Therapy · doi:10.1016/j.ymthe.2003.09.019 · Cited by 50
Gene Transfer into the Lung by Nanoparticle Dextran-Spermine/Plasmid DNA Complexes
Abdullah, S., Wendy-Yeo, W. Y., Hosseinkhani, H., Hosseinkhani, M., Masrawa, E., Ramasamy, R., Rosli, R., Rahman, S. A., & Domb, A. J. (2010) · BioMed Research International · doi:10.1155/2010/284840 · Cited by 53
The development of gene therapy for diseases of the lung
Gill, D. R., Davies, L. A., Pringle, I. A., & Hyde, S. C. (2004) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-003-3317-z · Cited by 50
Self-citation
Optimising non-viral gene delivery in a tumour spheroid model
Mellor, H. R., Davies, L. A., Caspar, H., Pringle, C. R., Hyde, S. C., Gill, D. R., & Callaghan, R. (2006) · The Journal of Gene Medicine · doi:10.1002/jgm.947 · Cited by 49
Self-citation
Plasmid DNA Production for Therapeutic Applications
Lara, A. R., & Ramı́rez, O. T. (2011) · Methods in molecular biology · doi:10.1007/978-1-61779-433-9_14 · Cited by 49
Hypoxia-specific gene expression for ischemic disease gene therapy
Kim, H., Mahato, R. I., & Lee, M. (2009) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2009.04.009 · Cited by 48
Increased Maintenance and Persistence of Transgenes by Excision of Expression Cassettes from Plasmid Sequences In Vivo
Riu, E., Grimm, D., Huang, Z., & Kay, M. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.558 · Cited by 54
Nucleic-acid based gene therapeutics: delivery challenges and modular design of nonviral gene carriers and expression cassettes to overcome intracellular barriers for sustained targeted expression
Hsu, C. Y. M., & Uludağ, H. (2012) · Journal of drug targeting · doi:10.3109/1061186x.2012.655247 · Cited by 47
Electroporation-mediated transfer of plasmids to the lung results in reduced TLR9 signaling and inflammation
Zhou, R., Norton, J. E., Zhang, N., & Dean, D. A. (2007) · Gene Therapy · doi:10.1038/sj.gt.3302936 · Cited by 46
The use of CpG-free plasmids to mediate persistent gene expression following repeated aerosol delivery of pDNA/PEI complexes
Davies, L. A., Hyde, S. C., Nunez-Alonso, G. A., Bazzani, R. P., Harding-Smith, R., Pringle, I. A., Lawton, A. E., Abdullah, S., Roberts, T. C., McCormick, D., et al. (2012) · Biomaterials · doi:10.1016/j.biomaterials.2012.04.019 · Cited by 45
Self-citation
A Comparative Analysis of Constitutive Promoters Located in Adeno-Associated Viral Vectors
Damdindorj, L., Karnan, S., Ota, A., Hossain, E., Konishi, Y., Hosokawa, Y., & Konishi, H. (2014) · PLoS ONE · doi:10.1371/journal.pone.0106472 · Cited by 54
Duration of Expression and Activity of Sleeping Beauty Transposase in mouse liver following hydrodynamic DNA delivery
Bell, J. B., Aronovich, E. L., Schreifels, J. M., Beadnell, T. C., & Hackett, P. B. (2010) · Molecular Therapy · doi:10.1038/mt.2010.152 · Cited by 45
Controlling the kinetics of transgene expression by plasmid design
Yew, N. S. (2005) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2004.12.009 · Cited by 43
Electroporation enhances reporter gene expression following delivery of naked plasmid DNA to the lung
Pringle, I. A., McLachlan, G., Collie, D., Jones, S., Lawton, A. E., Tennant, P. A., Baker, A., Gordon, C., Blundell, R. J., Varathalingam, A., et al. (2007) · The Journal of Gene Medicine · doi:10.1002/jgm.1026 · Cited by 43
Self-citation
Thioredoxin prevents the development and progression of elastase-induced emphysema
Kinoshita, T., Hoshino, T., Imaoka, H., Ichiki, H., Okamoto, M., Kawayama, T., Yodoi, J., Kato, S., & Aizawa, H. (2007) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2007.01.053 · Cited by 43
Nonionic Amphiphilic Block Copolymers Promote Gene Transfer to the Lung
Desigaux, L., Gourden, C., Bello-Roufaı̈, M., Richard, P., Oudrhiri, N., Lehn, P., Escande, D., Pollard, H., & Pitard, B. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.821 · Cited by 43
Non-viral vectors in cystic fibrosis gene therapy: recent developments and future prospects
Pringle, I. A., Hyde, S. C., & Gill, D. R. (2009) · Expert Opinion on Biological Therapy · doi:10.1517/14712590903055029 · Cited by 44
Self-citation
Efficient in vivo transfection and safety profile of a CpG-free and codon optimized luciferase plasmid using a cationic lipophosphoramidate in a multiple intravenous administration procedure
Lindberg, M. F., Gall, T. L., Carmoy, N., Berchel, M., Hyde, S. C., Gill, D. R., Jaffrès, P., Lehn, P., & Montier, T. (2015) · Biomaterials · doi:10.1016/j.biomaterials.2015.04.024 · Cited by 41
Self-citation
In vivo electroporation and ubiquitin promoter – a protocol for sustained gene expression in the lung
Gazdhar, A., Bilici, M., Pieróg, J., Ayuni, E., Gugger, M., Wetterwald, A., Cecchini, M. G., & Schmid, R. A. (2006) · The Journal of Gene Medicine · doi:10.1002/jgm.911 · Cited by 41
Detection of plasmid DNA vectors following gene transfer to the murine airways
Pringle, I. A., Raman, S., Sharp, W. W., Cheng, S. H., Hyde, S. C., & Gill, D. R. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302518 · Cited by 41
Self-citation
Adeno‐associated virus gene transfer in Morquio A disease – effect of promoters and sulfatase‐modifying factor 1
Alméciga-Díaz, C. J., Montaño, A. M., Tomatsu, S., & Barrera, L. A. (2010) · FEBS Journal · doi:10.1111/j.1742-4658.2010.07769.x · Cited by 41
Electrohydrodynamic Comminution: A Novel Technique for the Aerosolisation of Plasmid DNA
Davies, L. A., Hannavy, K., Davies, N., Pirrie, A., Coffee, R., Hyde, S. C., & Gill, D. R. (2005) · Pharmaceutical Research · doi:10.1007/s11095-005-5268-6 · Cited by 40
Self-citation
Dry powder aerosols of polyethylenimine (PEI)-based gene vectors mediate efficient gene delivery to the lung
Pfeifer, C., Hasenpusch, G., Üzgün, S., Aneja, M. K., Reinhardt, D., Kirch, J., Schneider, M., Claus, S., Frieß, W., & Rudolph, C. (2011) · Journal of Controlled Release · doi:10.1016/j.jconrel.2011.05.006 · Cited by 39
HIV Infects Bronchial Epithelium and Suppresses Components of the Mucociliary Clearance Apparatus
Chinnapaiyan, S., Parira, T., Dutta, R. K., Agudelo, M., Morris, A. M., Nair, M., & Unwalla, H. J. (2017) · PLoS ONE · doi:10.1371/journal.pone.0169161 · Cited by 39
Electroporation of the Vasculature and the Lung
Dean, D. A. (2003) · DNA and Cell Biology · doi:10.1089/104454903322625000 · Cited by 39
Expression of CFTR from a ciliated cell-specific promoter is ineffective at correcting nasal potential difference in CF mice
Ostrowski, L. E., Yin, W., Diggs, P. S., Rogers, T. D., O’Neal, W. K., & Grubb, B. R. (2007) · Gene Therapy · doi:10.1038/sj.gt.3302994 · Cited by 37
Optimisation of real-time quantitative RT-PCR for the evaluation of non-viral mediated gene transfer to the airways
Rose, A. C., Goddard, C. A., Colledge, W. H., Cheng, S. H., Gill, D. R., & Hyde, S. C. (2002) · Gene Therapy · doi:10.1038/sj.gt.3301792 · Cited by 37
Self-citation
Role for Centromeric Heterochromatin and PML Nuclear Bodies in the Cellular Response to Foreign DNA
Bishop, C. L., Ramalho, M., Nadkarni, N. A., Kong, W. M., Higgins, C. F., & Krauzewicz, N. (2006) · Molecular and Cellular Biology · doi:10.1128/mcb.26.7.2583-2594.2006 · Cited by 36
Self-citation
Long-term persistence of gene expression from adeno-associated virus serotype 5 in the mouse airways
Jones, S., Davies, L. A., Varathalingam, A., Gill, D. R., & Hyde, S. C. (2006) · Gene Therapy · doi:10.1038/sj.gt.3302815 · Cited by 36
Self-citation
High-Frequency Epigenetic Repression and Silencing of Retroviruses Can Be Antagonized by Histone Deacetylase Inhibitors and Transcriptional Activators, but Uniform Reactivation in Cell Clones Is Restricted by Additional Mechanisms
Katz, R. A., Jack-Scott, E., Narezkina, A., Palagin, I. S., Boimel, P. J., Kulkosky, J., Nicolas, É., Greger, J., & Skalka, A. M. (2007) · Journal of Virology · doi:10.1128/jvi.01643-06 · Cited by 36
Non-viral gene transfer therapy for cystic fibrosis
Ziady, A. G., Davis, P. B., & Konstan, M. W. (2003) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.3.3.449 · Cited by 37

Authors: Gill, D., Smyth, S., Goddard, C., Pringle, I., Higgins, C., Colledge, W., & Hyde, S.
Affiliations: John Radcliffe Hospital (GB)
University of Cambridge (GB)
Hammersmith Hospital (GB)
Venue: Gene Therapy
Publisher: Springer Science and Business Media LLC
Published: 2001-10-01
Volume: 8
Issue: 20
Pages: 1539-1546
ISSN: 0969-7128 (ISSN-L)
1476-5462
DOI: 10.1038/sj.gt.3301561
OpenAlex: W2122003649
Cited by: 214
References: 34
Citations by year: 2026: 6 · 2025: 4 · 2024: 1 · 2023: 1 · 2022: 2 · 2021: 2 · 2020: 3 · 2019: 4 · 2018: 3 · 2017: 6 · 2016: 4 · 2015: 6 · 2014: 9 · 2013: 7 · 2012: 12
FWCI: 3.61
Type: journal article
Language: en
Open access: closed
Licence: www.springer.com/tdm (tdm)
www.springer.com/tdm (vor)
Locations: Gene Therapy (publishedVersion)
PubMed (publishedVersion)
Funders: Wellcome Trust
Cystic Fibrosis Trust
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Animal Genetics and Reproduction
Keywords: persistent transgene expression, pulmonary gene therapy, promoter selection, cystic fibrosis, promoter silencing, reporter gene expression
MeSH terms: Administration, Inhalation, Animals, Avian Sarcoma Viruses, Cell Line, Cystic Fibrosis, Cytomegalovirus, Female, Gene Expression Regulation, Genetic Vectors, Humans, Luciferases, Lung, Mice, Inbred BALB C, Plasmids, Promoter Regions, Genetic, Simian virus 40, Time Factors, Genetic Therapy, Statistics, Nonparametric, Transgenes, Peptide Elongation Factor 1, Models, Animal, Ubiquitin C, Mice

About these checks

Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.

Statistics and arithmetic are recomputed, not judged.

Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.

What counts as a claim ›How we measure confidence ›How we check this ›

Are you an author, or do you see a problem with this page? Request a review

Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor1α promoter

Abstract

Overview

Integrity signals

Verification

Claims the paper makes

Claims other papers make about this one

Citations

Cites

Cited by

Related

Metadata

Record

About these checks